ALPHA 1-ANTITRYPSIN DEFICIENCY
Clinical trials for ALPHA 1-ANTITRYPSIN DEFICIENCY explained in plain language.
Never miss a new study
Get alerted when new ALPHA 1-ANTITRYPSIN DEFICIENCY trials appear
Sign up with your email to follow new studies for ALPHA 1-ANTITRYPSIN DEFICIENCY, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Gene therapy trial aims to fix protein deficiency behind lung and liver disease
Disease control Recruiting nowThis early-stage study is testing a new gene therapy called BEAM-302 in adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause lung and liver disease. The goal is to find a safe dose and see if the treatment can increase levels of a missing protective prot…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Beam Therapeutics Inc. • Aim: Disease control
Last updated Apr 03, 2026 21:10 UTC
-
First patients dosed in groundbreaking Alpha-1 therapy trial
Disease control Recruiting nowThis is the first study in people to test a new drug called AIR-001 for Alpha-1 Antitrypsin Deficiency (AATD). The main goal is to see if the treatment, given as an injection under the skin, is safe and how the body processes it. The study will enroll 54 adults with a specific ge…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: AIRNA Corporation • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
-
Daily inhaler trial aims to slow genetic lung damage
Disease control Recruiting nowThis study is testing whether inhaling a protein called AAT daily can slow down lung damage in adults with a genetic condition called Alpha-1 antitrypsin deficiency. For the first two years, half the participants will receive the active treatment and half will receive a placebo, …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE3 • Sponsor: Kamada, Ltd. • Aim: Disease control
Last updated Apr 03, 2026 14:41 UTC
-
Extended trial tests new therapy for genetic lung disease
Disease control Recruiting nowThis study aims to evaluate the long-term safety and effectiveness of an investigational drug called SAR447537 (INBRX-101) for adults with emphysema caused by Alpha-1 antitrypsin deficiency. The trial will follow 185 participants who continue receiving the treatment to monitor si…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Apr 02, 2026 14:56 UTC
-
First pill for genetic lung & liver disease enters human testing
Disease control Recruiting nowThis is a very early study to check if a new oral drug called BMN 349 is safe for people with Alpha-1 Antitrypsin Deficiency, a genetic condition that can damage the lungs and liver. A small group of 12 adults with specific genetic types of the disease will take a single dose of …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Mar 30, 2026 14:34 UTC
-
One-Shot gene therapy tested for rare lung disease
Disease control Recruiting nowThis is an early safety study for a new gene therapy designed to treat Alpha-1 Antitrypsin Deficiency, a genetic condition that can cause serious lung disease. Researchers are testing a single injection that aims to help the body produce a protective protein long-term. The study …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
-
First human tests begin for new genetic disease therapy
Disease control Recruiting nowThis early-stage study is testing a new drug called WVE-006 for people with Alpha-1 Antitrypsin Deficiency (AATD), a genetic condition that can damage the lungs and liver. Researchers will give the drug to 24 participants to first check if it's safe and how the body handles it. T…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Phase: PHASE1, PHASE2 • Sponsor: Wave Life Sciences Ltd. • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
-
Scientists probe immune cell failure in genetic lung diseases
Knowledge-focused Recruiting nowThis study aims to understand how two genetic lung diseases, Alpha-1 Antitrypsin Deficiency and Cystic Fibrosis, affect the function of a specific type of white blood cell in the lungs called a macrophage. Researchers will recruit 220 adults with these conditions to collect blood…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Apr 03, 2026 21:10 UTC
-
Hunting for early warning signs in a hidden lung disease
Knowledge-focused Recruiting nowThis study aims to find better ways to track lung disease in people with a specific genetic form of Alpha-1 antitrypsin deficiency (called PiMZ). Researchers will follow 80 participants for three years, using CT scans and blood tests to look for early signs that the lung disease …
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: Columbia University • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:41 UTC
-
Massive 50,000-Person study unlocks secrets of rare genetic disorder
Knowledge-focused Recruiting nowThis study aims to understand the real-world use and impact of genetic testing for Alpha-1 Antitrypsin Deficiency, a rare inherited condition. It will enroll 50,000 people at risk for the disease to learn about who gets tested, why, and what happens afterward. The research focuse…
Matched conditions: ALPHA 1-ANTITRYPSIN DEFICIENCY
Sponsor: University of Florida • Aim: Knowledge-focused
Last updated Mar 19, 2026 14:55 UTC